Medhavi Gupta, O. Akhtar, B. Bahl, Angel Mier-Hicks, Kristopher Attwood, Kayla Catalfamo, B. Gyawali, P. Torka
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引用次数: 0
摘要
与健康相关的生活质量(HRQoL)结果对于临床和政策决策非常重要。这项回顾性队列研究分析了2015年7月至2020年5月期间获得FDA批准的癌症药物试验的HRQoL数据。研究分析了纳入试验中报告 HRQoL 的比例、FDA 批准与首次报告 HRQoL 数据之间的延迟时间、HRQoL 结果及其与 OS(总生存期)和 PFS(无进展生存期)的相关性。在与 207 项 FDA 批准相关的 233 项试验中,50% 的试验报告了 HRQoL,其中只有 42% 的试验在 FDA 批准时报告了数据。2015年至2020年间,报告HRQoL的频率没有变化。只有30%的试验在主要出版物中首次报告了HRQoL数据。在115项提供HRQoL数据的试验中,43%的试验HRQoL有所改善,53%的试验保持稳定,4%的试验HRQoL恶化。在根据替代终点获得 FDA 批准的试验中(79%),45% 的试验报告了 HRQoL,只有 18% 的试验报告了 HRQoL 的改善。在获得FDA批准的试验中,HRQoL的报告率并不理想,在2015年至2020年间未见改善。HRQoL报告往往被延迟,且未在主要出版物中介绍。在根据替代终点获得批准的试验中,HRQoL报告更为稀少,只有少数试验的HRQoL有所改善。
Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology
Health-related quality of life (HRQoL) outcomes are important in making clinical and policy decisions. This study aimed to examine the HRQoL reporting in cancer drug trials leading to Food and Drug Administration (FDA) approvals.This retrospective cohort study analysed HRQoL data for trials leading to FDA approvals between July 2015 and May 2020. Proportion of included trials that reported HRQoL, latency between FDA approval and first report of HRQoL data, HRQoL outcomes, and their correlation with OS (overall survival) and PFS (progression-free survival) were analysed.Of the 233 trials associated with 207 FDA approvals, HRQoL was reported in 50% of trials, of which only 42% had the data reported by the time of FDA approval. There were no changes in frequency of HRQoL reporting between 2015 and 2020. HRQoL data were first reported in the primary publication in only 30% trials. Of the 115 trials with HRQoL data available, HRQoL improved in 43%, remained stable in 53% and worsened in 4% of trials. Among the trials that led to FDA approvals based on surrogate endpoints (79%), HRQoL was reported in 45% and improved only in 18% trials. There was no association between OS and PFS benefit and HRQoL outcomes.Rates of HRQoL reporting were suboptimal in trials that led to FDA approvals with no improvements seen between 2015 and 2020. HRQoL reporting was often delayed and not presented in the primary publication. HRQoL reporting was further sparse in trials with approvals based on surrogate endpoints and HRQoL improved in only a minority of them.